Efficacy will be determined/summarized by percent changes of the domain scores for upper extremity function, dexterity, mobility, pain, and self-care and functional ability testing from baseline. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]

Upper extremity function will be tested using the Grip-Pinch Test.

Dexterity will be tested using the Functional Dexterity Test.

Mobility will be tested using the 25 Foot Walk test

Pain will be determined by the Brief Pain Inventory-Short Form questionnaire or the Adolescent Pediatric Pain Tool

Change in respiratory function using a summarized analysis of the percentage change in FET, FIVC, FVC, FEV1, MVV and optional TLC values from the Baseline visit. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]

Timepoints: Baseline, Weeks: 24, 48, 96, 144 / ETV

Change in urinary KS over time as determined by descriptive statistics. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]

Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 144 weeks.

Drug: BMN 110

Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.

Other Names:

N-acetylgalactosamine-6-sulfatase

N-acetylgalactosamine-6-sulfate

sulfatase

galactose-6-sulfatase

GALNS

enzyme replacement therapy

ERT

Detailed Description:

Effect is defined by the following domains:

Upper extremity function and dexterity

Mobility

Pain

Self care and functional abilities

Eligibility

Ages Eligible for Study:

5 Years and older

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Is willing and able to provide written, signed informed consent (or their legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure. Patients who do not meet country and local age requirements for informed consent must be willing and able to provide written assent after the nature of the study has been explained and prior to performance of any research-related procedure.

Has documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA.

Is ≥ 5 years of age.

If sexually active, is willing to use an acceptable method of contraception while participating in the study.

Females of childbearing potential must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.

Is willing and able to perform all study procedures as physically possible.

Exclusion Criteria:

Is able to walk farther than a specified distance as assessed by the 6MWT.

Has previous hematopoietic stem cell transplant (HSCT).

Has received previous treatment with BMN 110.

Has a known hypersensitivity to any of the components of BMN 110.

Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the first 24 weeks of the study.

Has used any other investigational product or investigational medical device within 30 days prior to the Screening Visit or requires any investigational agent prior to completion of all scheduled study assessments.

Is pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study.

Has a concurrent disease or condition, including but not limited to symptomatic cervical spine instability or severe cardiac disease or complete paralysis due to a spinal cord injury (defined as an inability to move arms and legs), that would interfere with study participation or safety as determined by the Investigator.

Has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01697319